enGene (ENGN) Jefferies 2024 Global Healthcare Conference summary

Vision and platform differentiation

• Aims to mainstream genetic medicines using a proprietary, non-viral platform with broad applications beyond bladder cancer.

• EG-70 offers single-step, patient-friendly administration, activating both innate and adaptive immunity.

• Platform enables scalable, low-cost manufacturing and avoids complex handling of viral therapies.

• Unlocks mucosal tissues for genetic medicine delivery, expanding potential indications.

Clinical development and data

• Phase I study met all endpoints, showing 73% complete response (CR) at any time and strong durability.

• Phase II registrational trial enrolling 100 patients, with interim data expected mid-2024 and BLA submission planned for Q1 2026.

• Safety profile is favorable, with most adverse events being mild and consistent with bladder instrumentation.

• No anti-drug antibodies generated, allowing flexible dosing and repeat administration.

Market opportunity and competitive landscape

• Addresses high-risk, non-muscle invasive bladder cancer (NMIBC) patients unresponsive to BCG, a multibillion-dollar market with high unmet need.

• EG-70 designed for community urology clinics, offering workflow advantages over competitors like BCG, Adstiladrin, and TAR-200.

• Fast Track designation received; additional oncology indications and BCG-naïve cohort data expected.

• Market measured by prevalence due to patient preference to avoid radical cystectomy.